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PHYTIC ACID-BASED TERNARY COMPLEX BIONIC NANO MATERIAL, AND PREPARATION METHOD AND USE THEREOF

Resumen de: EP4706690A1

The present application relates to the field of biological medicines, in particular to a phytic acid-based ternary complex bionic nano material. The ternary complex bionic nano material is composed of phytic acid, metal ions and a protein/polypeptide. The ternary complex is formed by connecting the protein/polypeptide with phytic acid using metal cations as a cation bridge. According to the application, the phytic acid ternary complex having different protein/polypeptide groups is synthesized by using various metal ions as bridges. A novel nano diagnosis and treatment probe having high biocompatibility is constructed, which can be reassembled and gathered for a long time at a tumor part, and has coordination-loaded imaging/therapeutic metal ions. The functions of long-acting tumor imaging, treatment or integrated diagnosis and treatment can be achieved, and the solution can be expected to be used as a novel bionic nano material in the field of biomedicine. In addition, the preparation process of the nano material is simple, and industrial production is easy to implement.

A PROIMMUNOTOXIN THERAPEUTIC NANOPLATFORM AGAINST CANCER

Resumen de: WO2024229147A2

Aspects of the disclosure relate to compositions comprising encapsulated immunotoxins and methods for using the encapsulated immunotoxins. The encapsulated immunotoxins may be capable of reaching otherwise difficult to target tissues, such as the brain, and may resist denaturation and degradation prior to reaching the targeted tissue.

CORTICOSTEROID NANOSUSPENSIONS AND USES THEREOF

Resumen de: MX2025013065A

Suspension formulations of nanoparticles of clobetasol propionate are described. The suspensions can be used therapeutically to treat skin and ocular burns; to enhance wound healing; to prevent or reduce hypertrophic scarring/keloids; to treat allergic rhinitis/sinusitis, asthma, inner ear disorders including hearing loss, tinnitus, or vertigo, tenosynovitis, tendinitis, enthesitis or arthritis.

PEGYLATED RECONSTITUTED HIGH-DENSITY LIPOPROTEIN NANOPARTICLES

Resumen de: EP4706645A1

The present invention relates to PEGylated reconstituted high-density lipoprotein nanoparticles having the effect of preventing or treating neurodegenerative diseases. Specifically, the present invention relates to nanoparticles and a method for producing the same, a phospholipid layer of the produced nanoparticles being protected by PEG due to PEG-lipid or a derivative thereof being included in the process of preparing a fluid comprising a hydrophobic material and a fluid comprising a hydrophilic material. The PEGylated nanoparticles have the ability to avoid the rejection mechanism of the immune response in the body while maintaining the existing transport ability across the blood-brain barrier, thereby having excellent stability and exhibiting long-term pharmacological effect due to high circulation ability in the body, and thus can be effectively utilized as a drug or a drug carrier.

RECONSTITUTED HIGH-DENSITY LIPOPROTEIN NANOPARTICLES COMPRISING CHOLESTEROL

Resumen de: EP4706644A1

The present invention relates to: reconstituted high-density lipoprotein nanoparticles comprising cholesterol; and a composition for preventing or treating Alzheimer's disease and cancer, the composition comprising the nanoparticles. Specifically, the reconstituted high-density lipoprotein nanoparticles comprising cholesterol according to the present invention have an excellent cell influx rate and promote cholesterol efflux from cells, thus having a cancer cell killing effect, and can therefore be used for preventing or treating cancer.

PHARMACEUTICAL COMPOSITION COMPRISING TELOMERASE ACTIVATOR AND NANOPARTICLES FOR DRUG DELIVERY, AND COMPOSITION CONTAINING SAME FOR PREVENTION, ALLEVIATION, OR TREATMENT OF HAIR LOSS

Resumen de: EP4706660A1

The present invention relates to a pharmaceutical composition comprising a telomerase activator and nanoparticles for drug delivery and a composition containing same for the prevention, alleviation, or treatment of hair loss. More specifically, the present invention relates to a composition effective for promoting the regeneration and growth of hair follicles, the composition being a nanoliposome, nano-liposome, nano-silica particle, or nano-bubble composition in which drugs such as the plant extract component TA-65 and a derivative thereof that increase telomerase activity, and the telomerase activating compound GPC and a derivative thereof are encapsulated

がん治療用のインターロイキン－２およびインターロイキン－１２

Resumen de: WO2024182707A1

The present disclosure provides a first polynucleotide (e.g., a recombinant or isolated first polynucleotide) encoding an Interleukin (IL)-2 polypeptide and a second polynucleotide (e.g., a recombinant or isolated second polynucleotide) encoding an IL-12 polypeptide; recombinant nucleic acids and/or gene delivery vehicles comprising the same; compositions, formulations, and medicaments comprising the one or more of the polynucleotides, recombinant nucleic acids, and/or gene delivery vehicles; methods of their use (e.g., for the treatment of cancer); and articles of manufacture or kits thereof.

一种环状RNA用于制备核酸药物的应用

Resumen de: CN121622725A

本发明涉及一种环状RNA用于制备核酸药物的应用。本发明经实验研究证实，环状 RNA circPde5a 可用于制备长效减肥降糖核酸药物；将 circPde5a 递送至靶组织（如胰岛组织、白色脂肪组织）后，可通过调控靶组织代谢相关通路，实现减肥与降糖的双重效果。同时，该 circPde5a 还可与现有治疗手段协同使用。

免疫優性Ｔ細胞エピトープを欠く組換えアデノ随伴ウイルスベクターおよびその使用

Resumen de: WO2024178113A1

Recombinant adeno-associated virus (AAV) vectors encoding a modified VP1 protein lacking an immunodominant T cell epitope, as well as AAV vector particles containing the modified VP1 protein, are described. Use of the recombinant AAV vectors and vector particles as improved gene therapy vectors with reduced immunogenicity is also described. Isolated VP1 peptides containing an immunodominant T cell epitope, and use thereof, is further described.

一种自组装纳米粒子、制备方法及应用

Resumen de: CN121622613A

本发明属于纳米材料和生物医药技术领域，涉及一种无载体自组装药物纳米粒子及其制备方法和应用。本发明提供的无载体自组装药物纳米粒子由化疗药物、光敏剂、生物矿化外壳及癌细胞膜四种组分自组装而成；其中，化疗药物为盐酸阿霉素，光敏剂为吲哚菁绿，生物矿化外壳为高锰酸钾溶液制备得到，癌细胞膜为4T1癌细胞膜。本发明还提供了制备这种纳米粒子的方法，以及包含这种纳米粒子的药物及纳米粒子在制备肿瘤治疗药物中的应用。本发明提供的纳米粒子具有载药率高、制备简单、无载体的特点，体外细胞实验和动物实验证实，该纳米粒子具有显著的肿瘤治疗效果。

编码胶原蛋白的核酸分子及其用途

Resumen de: WO2026046285A1

Provided are an mRNA comprising a nucleotide sequence encoding collagen, and a composition comprising the mRNA and a delivery vector. An mRNA molecule is introduced into a subject by means of the composition, which can be used for improving the skin condition.

环状单链DNA递送载体及其应用

Resumen de: CN121622609A

本发明公开了一种环状单链DNA递送载体及其应用，属于药物制剂技术领域，该环状单链DNA递送载体包括可电离阳离子脂质、脂质聚乙二醇缀合物、甾醇类化合物和1‑硬脂酰基‑2‑油酰基卵磷脂；以环状单链DNA递送载体中存在的总脂质的总摩尔数为1计，可电离阳离子脂质的摩尔百分比为25％‑60％，脂质聚乙二醇缀合物的摩尔百分比为1.0％‑3.5％，甾醇类化合物的摩尔百分比为20％‑50％，1‑硬脂酰基‑2‑油酰基卵磷脂的摩尔百分比为5％‑30％。该递送载体在N/P比为4‑10：1的条件下递送环状单链DNA展现出了卓越的转染效率，在基因治疗领域具有广泛的应用前景。

一种金银花总提取物吸入溶液及其制备方法和应用

Resumen de: CN121622624A

本发明属于生物医药技术领域，具体涉及一种金银花总提取物吸入溶液及其制备方法和应用。本发明创新性地将水溶性差的金银花总提取物与磷脂、壳聚糖定向组装成结构均一的壳聚糖‑磷脂纳米粒，并采用微流控技术制备纳米粒。该策略显著提高了金银花总提取物的水溶性与稳定性，并基于此纳米粒分散体系，成功制备了适于肺部给药的吸入溶液。所获得的制剂具有物理稳定性高、生物利用度潜力佳的特点。本发明提供的金银花总提取物吸入溶液能够通过显著抑制肺部炎症反应、减轻肺水肿及保护肺泡结构完整性等多重机制，有效缓解脓毒性肺炎的病理进程。特别是其高剂量组展现出的优于阳性对照药的疗效，结合良好的剂量效应关系，表明该制剂在脓毒性肺炎治疗中具有显著的开发潜力和临床转化前景。

治療薬の生体内分布および有効性の受容体媒介制御のための標的

Resumen de: CN120435500A

Disclosed herein, including novel BBB spanning receptors on the blood brain barrier (BBB) interface, targeting peptides and derivatives thereof capable of binding to these novel receptors, and related methods of using these receptors to increase permeability of BBB and deliver agents to the nervous system (e.g., CNS). In some embodiments, the BBB is LRP6 across the receptor. Also disclosed herein are recombinant adeno-associated viruses (rAAVs) with increased specificity and transduction efficiency across BBB, as well as related compositions and methods of treating various diseases and conditions.

疾患の処置のためのＫＲＡＳ阻害のための組成物および方法

Resumen de: MX2025009845A

The present disclosure provides pharmaceutical compositions comprising a peptide- polynucleotide complex, wherein the peptide comprises an amino acid sequence with at least 80%, at least 85%, at least 90%, at least 95%, at least 98%, at least 99%, or 100% identity to the amino acid sequence of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3; and wherein the polynucleotide is a small interfering RNA (siRNA) targeting human KRAS mRNA, wherein the target sequence of human KRAS mRNA does not encode G12, G13, or Q61 with reference to SEQ ID NO: 4 or a mutant amino acid at position 12, 13, or 61 with reference to SEQ ID NO: 4.

アジュバントを全く含まないカチオン性多糖ナノ粒子を介して不活性化全細菌を送達するための系を含むワクチン組成物

Resumen de: CN120712079A

The present invention relates to the field of vaccine compositions. The invention more particularly relates to a prophylactic vaccine composition comprising killed intact bacteria intended for use in mammals and birds, said bacteria being wrapped with a cationic agent, in particular cationic nanoparticles.

用于脂质纳米颗粒的脂质

Resumen de: US2024423915A1

Compounds are provided having the following Formula (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3, G1, G2, L1, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

用于靶向治疗具核梭杆菌浸润的结直肠癌的纳米药物体系及其制备方法

Resumen de: CN121622605A

本发明公开了用于靶向治疗具核梭杆菌浸润的结直肠癌的纳米药物体系及其制备方法。本发明属于生物技术领域，具体涉及用于靶向治疗具核梭杆菌浸润的结直肠癌的纳米药物体系及其制备方法。本发明的具有抑制肿瘤细胞活性或抗肿瘤活性的药物活性成分含有NM@PLGA‑MTI‑OXA，NM@PLGA‑MTI‑OXA由中性粒细胞膜囊泡和PLAG‑MTI‑OXA组成，其中PLAG‑MTI‑OXA是由聚乳酸‑羟基乙酸共聚物负载化疗药物MTI及OXA得到的纳米颗粒，中性粒细胞膜囊泡来源于骨髓中性粒细胞。本发明开发的NM@PLGA‑MTI‑OXA能够有效靶向具核梭杆菌浸润的结直肠组织，成功实现抗菌抗肿瘤效果，具有应用价值。

可离子化硫脂质及其用途

Resumen de: AU2024315222A1

The present disclosure provides a compound of formula (I): (I), or a pharmaceutically acceptable salt thereof, that is useful for forming particles (e.g., lipid nanoparticles) for delivery of nucleic acids. The present disclosure further provides particle compositions comprising the compound of formula I, as well as uses thereof.

一种狂犬病毒G蛋白mRNA疫苗冻干制剂的制备及应用

Resumen de: CN121628927A

本发明公开了一种狂犬病毒G蛋白mRNA疫苗冻干制剂的制备及应用，涉及兽用生物制品领域。本发明通过体外转录技术规模化制备狂犬病毒G蛋白mRNA，并采用微流控包封工艺成功构建mRNA‑LNP（脂质纳米颗粒）递送系统。通过冻干保护剂配方筛选（涵盖蔗糖、海藻糖及甘露醇）及冻干程序参数优化（包括预冻温度、主干燥速率及二次干燥残留水分控制），最终获得可在4℃条件下稳定存在的冻干mRNA疫苗制剂。

一种新型佐剂多肽序列及其应用

Resumen de: CN121627848A

本发明涉及疫苗领域，特别涉及一种新型佐剂多肽序列及其应用。本发明提供的佐剂多肽序列包含破伤风类毒素表位P2、P16氨基酸序列，还包含P30和/或PX的氨基酸序列。该佐剂多肽序列通过增加表位多样性，旨在扩大免疫反应的覆盖群体。同时，本发明利用LNP(脂质纳米颗粒)递送系统，将编码佐剂多肽与初始抗原蛋白的mRNA高效导入体内，有效提升了初始抗原的免疫刺激能力，从而在减少初始抗原使用剂量的同时，仍能达到或超过传统剂量的免疫效果。进一步，本发明提供的佐剂多肽序列与初始抗原序列优选采用融合的方式，简化了核酸疫苗中mRNA的组成，降低了疫苗制备的复杂性，为疫苗工艺开发提供了新思路，具有显著的技术进步和临床应用潜力。

用于降低雷公藤甲素肝毒性的RNAi及其应用

Resumen de: CN121628906A

本发明公开了用于降低雷公藤甲素肝毒性的RNAi及其应用，属于生物医药技术领域。本发明将靶向 Cyp2e1 基因的 siRNA 经 2′‑O 甲基修饰及 dTsdT 3′硫代磷酸酯连接修饰后，通过脂质纳米颗粒（LNPs）递送。经静脉注射给药后，24h 内可使小鼠肝脏 Cyp2e1 蛋白表达下降 70% 以上，通过降低血清谷丙转氨酶 / 谷草转氨酶水平、抑制肝脏氧化应激、减少 TNF‑α/IL‑1β/IL‑6 等炎症因子释放，显著减轻雷公藤甲素诱导的亚急性肝损伤。本发明克服了现有减毒方案药效降低或靶向性欠佳的缺陷，提供了靶向调控效果显著、安全性高的临床辅助减毒技术方案，为雷公藤甲素的安全临床应用提供了新路径。

用于治疗结晶样视网膜变性的药物制剂

Resumen de: WO2025030426A1

Provided is a pharmaceutical preparation for treating Bietti's crystalline dystrophy (BCD), comprising a recombinant AAV expressing CYP4V2, sodium chloride, poloxamer, phosphate, and water for injection, and having a pH between 7.0 to 7.6. The pharmaceutical preparation has an excellent virus titer stability, very few AAV aggregates, a high mRNA expression of the target gene after multiple times of freezing and thawing, and/or an excellent high temperature stability. Moreover, the animal experiments in vivo demonstrate that the preparations with the formulations have an excellent property in reducing local inflammatory response of the retina, and further clinical trials demonstrate that the pharmaceutical preparations conform to the relevant technical specifications for safety and effectiveness in the Chinese Pharmacopoeia and the United States Pharmacopoeia (USP).

一种多巴胺类阳离子脂质化合物及其制备方法和应用

Resumen de: CN121627529A

本发明公开了一种多巴胺类阳离子脂质化合物及其制备方法和应用，属于有机合成技术领域。本发明以盐酸多巴胺为初始原料，随后与二碳酸二叔丁酯或氯甲酸叔丁酯进行亲加成核反应，然后在碱性催化条件下与1‑溴卤代烃反应，得到多巴胺衍生物；接着通过三氟乙酸将多巴胺衍生物上的叔丁氧羰基脱去，在碱性条件下与卤代烃反应，得到多巴胺类阳离子脂质化合物。本发明制备的多巴胺类阳离子脂质化合物能够增强生物活性分子和治疗分子的细胞内递送效率，将多巴胺类阳离子脂质化合物作为递送载体制备的药物制剂可将治疗有效剂量的生物活性分子递送至靶细胞处，具有潜在的应用价值。

顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法

Nº publicación: JP2026041765A 10/03/2026

Solicitante:

アビディティーバイオサイエンシーズ，インク．

Resumen de: US2025340880A1

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.